Cyclerion Therapeutics, Inc. · NASDAQ:CYCN
Cyclerion Therapeutics — UMDF Power Surge 2020
7 slides from Cyclerion Therapeutics's conference presentation, Jun 26, 2020
Slide 1: A Phase 2a pilot study evaluating IW-6463, a CNS-penetrant sGC stimulator, in individuals with MELAS
A Phase 2a pilot study evaluating IW-6463, a CNS- penetrant sGC stimulator, in individuals with MELAS Chad Glasser PharmD MPH Director, Clinical Research CONFIDENTIAL UMDF Power Surge – June 26th, 2020
Slide 2: Rationale for the evaluation of IW-6463, a CNS-penetrant sGC stimulator, in mitochondrial disease
Rationale for the evaluation of IW-6463, a CNS-penetrant sGC stimulator, in mitochondrial disease • NO-sGC-cGMP signaling plays a central role in mitochondrial biogenesis and function, neuroprotection, and vascular homeostasis • ~80% of individuals with mitochondrial disease have CNS symptoms • Nitr
Slide 3: In preclinical studies, IW-6463 demonstrates positive effects across four key domains of neurodegenerative disease
In preclinical studies, IW-6463 demonstrates positive effects across four key domains of neurodegenerative disease Improved cerebral blood flow in areas associated with memory and Cerebral Blood Flow arousal by fMRI BOLD imaging Cellular Increased ATP and restored an already depressed gene expressio
Slide 4: IW-6463 has the potential to restore NO signaling in individuals with mitochondrial disease
IW-6463 has the potential to restore NO signaling in individuals with mitochondrial disease Deficient NO signaling Restore NO signaling • Impaired neurovascular blood flow • Increase blood flow • Mitochondrial dysfunction • Improve bioenergetics • Neuroinflammation • Decrease inflammation • Neurodeg
Slide 5: IW-6463 Phase 1: CNS exposure, target engagement, PK, and safety
IW-6463 Phase 1: CNS exposure, target engagement, PK, and safety A three-part, randomized, placebo-controlled study in 110 healthy volunteers Phase 1 (completed) Overview of results • Well tolerated at all single and multi-dose levels • No discontinuations due to AEs, no SAEs Dose Selected • All AEs
Slide 6: MELAS: rare neurometabolic mitochondrial disease of high unmet need
MELAS: rare neurometabolic mitochondrial disease of high unmet need Genetically/phenotypically defined, nitric oxide deficiency, no approved treatments MELAS Symptom Overview Rationale for selection of MELAS Clinical precedence for NO-sGC-cGMP pathway Nervous system Seizures, SLEs, ataxia, L-Arginin
Slide 7: Phase 2a multicenter, open-label, pilot study in individuals with MELAS
Phase 2a multicenter, open-label, pilot study in individuals with MELAS Objectives Disease domain Assessment Evaluate safety, tolerability, and pharmacodynamics; assess near-term Mitochondrial Lactate*, GDF-15, pyruvate, impact on disease-specific biomarkers dysfunction alanine, IL1-β, L-arginine En